Related Experiment Video

Updated: Jun 26, 2026

Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study
08:00

Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study

Published on: April 11, 2018

Impact of Highly Effective CFTR Modulator Therapy on Physical Activity, Sleep, and Sinonasal Symptoms in Preschool

Stella Schellhorn1, Hanna Schmidt1, Ales Janda1

  • 1Department of Pediatrics and Adolescent Medicin, Ulm University Medical Center, Ulm University, Eythstrasse 24, 89075 Ulm, Germany.

Advances in Respiratory Medicine
|June 25, 2026
PubMed

Insights

Elexacaftor/Tezacaftor/Ivacaftor (ETI) therapy improved physical activity and parental perceptions in young cystic fibrosis (CF) patients. Improvements in physical activity may be seasonal, with sustained positive parental feedback observed.

Area of Science:

  • Pediatric Pulmonology
  • Cystic Fibrosis Therapeutics
  • CFTR Modulator Therapy

Background:

  • Elexacaftor/Tezacaftor/Ivacaftor (ETI) is a highly effective modulator for cystic fibrosis (CF).
  • Limited data exist on ETI's impact on physical activity, sleep, sinonasal symptoms, and parent-perceived outcomes in preschool-aged children with CF.
  • Understanding these effects is crucial for comprehensive CF management in young populations.

Purpose of the Study:

  • To evaluate the effects of ETI therapy on physical activity, sleep, sinonasal symptoms, and parental perceptions in preschool-aged children with CF.
  • To assess the safety and tolerability of ETI in this specific pediatric cohort.
  • To provide preliminary data for future larger-scale controlled studies.

Main Methods:

  • Prospective, observational, single-center cohort study including ten children (aged 2-6 years) with CF eligible for ETI.
  • Data collection via wrist-worn activity trackers and standardized questionnaires at baseline and 1, 3, 6, and 12 months post-ETI initiation.
  • Outcomes measured: step count, moderate-to-vigorous physical activity minutes, sleep parameters, sinonasal symptoms, and parental perceptions.

Main Results:

  • ETI was well-tolerated with significant reduction in sweat chloride levels.
  • Physical activity (steps, active minutes) improved at 3 and 6 months but returned to baseline by 12 months.
  • Parental perception of physical performance improved and was sustained; sleep parameters and sinonasal symptoms remained stable and low.

Conclusions:

  • ETI therapy is safe and well-tolerated in young children with CF.
  • Observed improvements in physical activity may be influenced by seasonality.
  • Sustained improvements in parental-reported outcomes and stable sleep patterns are encouraging, warranting further investigation in larger cohorts.

Keywords:
CFTR-modulatoractigraphyactivitycystic fibrosispreschool childrensinonasal symptomssleepwearables

More Related Videos

Standardized Measurement of Nasal Membrane Transepithelial Potential Difference (NPD)09:47

Standardized Measurement of Nasal Membrane Transepithelial Potential Difference (NPD)

Published on: September 13, 2018

Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients07:04

Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients

Published on: February 11, 2017

Related Concept Videos

Cystic Fibrosis: Management01:24

Cystic Fibrosis: Management

Cystic fibrosis (CF) is an autosomal recessive disorder that predominantly affects individuals of Northern European descent, occurring at a rate of 1 in 3500. It is caused by a genetic mutation in a gene on chromosome 7, most commonly the ΔF508 mutation, that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This results in thicker mucus secretions and obstruction pathologies in multiple organs, including the lungs and sinuses.
Sinus disease and chronic sinusitis...
Cystic Fibrosis: Pathogenesis01:23

Cystic Fibrosis: Pathogenesis

Cystic fibrosis (CF), an autosomal recessive disorder, significantly affects the function of exocrine glands. This genetically inherited disease is characterized by the production of thick and sticky mucus, which can severely affect various organs and systems in the body.
CF is primarily caused by a genetic mutation in a chromosome 7 gene coding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The most common gene mutation leading to CF is the ΔF508 mutation, but...
Chest Physiotherapy01:24

Chest Physiotherapy

Chest Physiotherapy (CPT) is a therapeutic technique used in respiratory care to improve ventilation, clear bronchial secretions, and enhance the efficiency of respiratory muscles. This therapy includes three primary procedures: postural drainage, percussion, and vibration. It can be performed on spontaneously breathing patients and those who are intubated and mechanically ventilated.
Purpose
CPT is primarily used for patients with excessive bronchial secretions who have difficulty clearing...